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10-Q

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, DC 20549

FORM 10-Q

(Mark One)


☒	QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2023


☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from __________________ to __________________

Commission File Number: 001-39979

VOR BIOPHARMA INC.

(Exact Name of Registrant as Specified in its Charter)


Delaware	81-1591163
(State or other jurisdiction of incorporation or organization)	(I.R.S. Employer Identification No.)
100 Cambridgepark Drive, Suite 101 Cambridge, Massachusetts	02140
(Address of principal executive offices)	(Zip Code)

Registrant’s telephone number, including area code: (617) 655-6580

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading
Symbol(s)

Name of each exchange on which registered

Common Stock, $0.0001 par value per share

VOR

Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

Number of shares of the registrant’s Common Stock outstanding as of November 3, 2023 was 67,805,687.

Table of Contents


		Page
PART I.	FINANCIAL INFORMATION	1

Item 1.	Financial Statements (Unaudited)	1
	Condensed Consolidated Balance Sheets as of September 30, 2023 and December 31, 2022	1
	Condensed Consolidated Statements of Operations and Comprehensive Loss for the Three and Nine Months Ended September 30, 2023 and 2022	2
	Condensed Consolidated Statements of Stockholders’ Equity for the Three and Nine Months Ended September 30, 2023 and 2022	3
	Condensed Consolidated Statements of Cash Flows for the Nine Months Ended September 30, 2023 and 2022	4
	Notes to Condensed Consolidated Financial Statements (Unaudited)	5
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	11
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	19
Item 4.	Controls and Procedures	19

PART II.	OTHER INFORMATION	20

Item 1.	Legal Proceedings	20
Item 1A.	Risk Factors	20
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	20
Item 6.	Exhibits	21
Signatures		22

Note Regarding Company References

Throughout this Quarterly Report on Form 10-Q, the “Company,” “Vor,” “Vor Bio,” “Vor Biopharma Inc.,” “we,” “us,” and “our,” except where the context requires otherwise, refer to Vor Biopharma Inc. and its consolidated subsidiary, and “our board of directors” refers to the board of directors of Vor Biopharma Inc.

Special Note Regarding Forward-Looking Statements

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans, and objectives of management, are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “might,” “intend,” “target,” “ongoing,” “project,” “estimate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions intended to identify statements about the future. These statements speak only as of the date of this Quarterly Report on Form 10-Q and involve known and unknown risks, uncertainties and other important factors that may cause our actual results, levels of activity, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.

We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition and results of operations. These forward-looking statements include, without limitation, statements about:

•

the timing, progress and results of our preclinical studies and clinical trials of our product candidates, including statements regarding the timing and pace of initiation, enrollment and completion of studies or trials and related preparatory work, the period during which the results of the trials will become available and plans with respect to our research and development programs;

•

the timing and success of our in-house or third-party clinical manufacturing capabilities and efforts;

•

the timing of any submission of filings for regulatory approval of, and our ability to obtain and maintain regulatory approvals for, our product candidates for any indication;

•

our ability to identify patients with the diseases treated by our product candidates, and to enroll patients in trials;

•

our expectations regarding the market acceptance and opportunity for and clinical utility of our product candidates, if approved for commercial use;

•

our expectations regarding the scope of any approved indication for any product candidate;

•

our ability to successfully commercialize our product candidates;

•

our estimates of our expenses, ongoing losses, future revenue, capital requirements and our need for or ability to obtain additional funding;

•

our ability to establish or maintain collaborations or strategic relationships;

•

our ability to identify, recruit and retain key personnel, including executive officers and members of management;

•

our reliance upon intellectual property licensed from third parties and our ability to obtain such licenses on commercially reasonable terms or at all;

•

our ability to protect and enforce our intellectual property position for our product candidates, and the scope of such protection;

•

our financial performance;

•

the period over which we estimate our existing cash, cash equivalents and marketable securities will be sufficient to fund our future operating expenses and capital expenditure requirements;

•

our competitive position and the development of and projections relating to our competitors or our industry;

•

the impact of laws and regulations; and

•

our expectations regarding the time during which we will be an emerging growth company under the Jumpstart Our Business Startups Act of 2012.

You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results may be materially different from what we

expect. The forward-looking statements contained in this Quarterly Report on Form 10-Q are made as of the date of this Quarterly Report on Form 10-Q, and we do not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. You should refer to the “Summary Risk Factors” and “Risk Factors” sections in our Annual Report on Form 10-K for the year ended December 31, 2022 for a discussion of material factors that could cause actual results or events to differ materially from the forward-looking statements that we make.

iii

PART I—FINANCIAL INFORMATION

Item 1. Financial Statements (Unaudited).

VOR BIOPHARMA INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(UNAUDITED)


	September 30,			December 31,
(in thousands, except share amounts)	2023			2022
Assets
Current assets:
Cash and cash equivalents	$	45,026		$	57,706
Marketable securities		115,072			172,539
Prepaid expenses		3,673			4,368
Other current assets		667			2,337
Total current assets		164,438			236,950
Restricted cash equivalents		2,413			2,413
Property and equipment, net		10,824			12,634
Operating lease right-of-use assets		41,262			44,444
Other assets		4,086			2,925
Total assets	$	223,023		$	299,366
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable	$	1,309		$	1,772
Accrued liabilities		10,274			7,889
Operating lease liabilities		3,743			3,272
Other current liabilities		62			186
Total current liabilities		15,388			13,119
Long-term liabilities:
Operating lease liabilities—non-current		32,854			35,640
Total liabilities		48,242			48,759
Stockholders’ equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized as of September 30, 2023 and December 31, 2022; 0 shares issued and outstanding as of September 30, 2023 and December 31, 2022		—			—
Common stock, $0.0001 par value; 400,000,000 shares authorized as of September 30, 2023 and December 31, 2022; 67,798,343 and 66,079,597 shares issued and 67,785,469 and 65,996,138 outstanding as of September 30, 2023 and December 31, 2022, respectively		7			7
Additional paid-in capital		488,873			473,587
Accumulated other comprehensive loss		(280	)		(770	)
Accumulated deficit		(313,819	)		(222,217	)
Total stockholders’ equity		174,781			250,607
Total liabilities and stockholders’ equity	$	223,023		$	299,366

The accompanying notes are an integral part of these condensed consolidated financial statements.

VOR BIOPHARMA INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(UNAUDITED)


	Three Months Ended September 30,						Nine Months Ended September 30,
(in thousands, except share and per share amounts)	2023			2022			2023			2022
Operating expenses:
Research and development	$	27,606		$	16,875		$	73,418		$	47,488
General and administrative		7,710			7,226			24,494			21,205
Total operating expenses	$	35,316		$	24,101		$	97,912		$	68,693
Loss from operations	$	(35,316	)	$	(24,101	)	$	(97,912	)	$	(68,693	)
Other income:
Interest income		2,126			313			6,310			509
Total other income		2,126			313			6,310			509
Net loss	$	(33,190	)	$	(23,788	)	$	(91,602	)	$	(68,184	)
Net loss per share attributable to common stockholders, basic and diluted	$	(0.49	)	$	(0.63	)	$	(1.37	)	$	(1.81	)
Weighted-average common shares outstanding, basic and diluted		67,607,713			38,009,022			66,973,771			37,582,463

Other comprehensive income (loss):
Unrealized gain (loss) on available for sale investments		156			148			490			(1,178	)
Total other comprehensive income (loss)		156			148			490			(1,178	)
Comprehensive loss	$	(33,034	)	$	(23,640	)	$	(91,112	)	$	(69,362	)

The accompanying notes are an integral part of these condensed consolidated financial statements.

Vor Biopharma Inc.

CONDENSED CONSOLIDATED Statements of stockholders’ EQUITY

(Unaudited)


	Common Stock				Additional Paid-In			Accumulated other comprehensive			Accumulated			Total Stockholders’ Equity
(in thousands, except share amounts)	Shares		Amount		Capital			loss			Deficit			(Deficit)
Balance at December 31, 2022		65,996,138	$	7	$	473,587		$	(770	)	$	(222,217	)	$	250,607
Issuance of common stock upon vesting of RSUs and exercise of stock options, net of shares withheld for taxes		205,485		—		(340	)		—			—			(340	)
Issuance of common stock from open market sales agreement		733,274		—		3,717			—			—			3,717
Stock-based compensation expense		—		—		4,068			—			—			4,068
Other comprehensive income		—		—		—			596			—			596
Net loss		—		—		—			—			(28,433	)		(28,433	)
Balance at March 31, 2023		66,934,897	$	7	$	481,032		$	(174	)	$	(250,650	)	$	230,215
Issuance of common stock upon vesting of RSUs, vesting and exercise of stock options, and issuance of common stock from ESSP, net of shares withheld for taxes		381,775		—		(317	)		—			—			(317	)
Issuance of common stock from open market sales agreement		116,888		—		555			—			—			555
Stock-based compensation expense		—		—		4,238			—			—			4,238
Other comprehensive loss		—		—		—			(262	)		—			(262	)
Net loss		—		—		—			—			(29,979	)		(29,979	)
Balance at June 30, 2023		67,433,560	$	7	$	485,508		$	(436	)	$	(280,629	)	$	204,450
Issuance of common stock upon vesting of RSUs, vesting and exercise of stock options, net of shares withheld for taxes		219,409		—		(183	)		—			—			(183	)
Issuance of common stock from open market sales agreement		132,500		—		386			—			—			386
Stock-based compensation expense		—		—		3,162			—			—			3,162
Other comprehensive loss		—		—		—			156			—			156
Net loss		—		—		—			—			(33,190	)		(33,190	)
Balance at September 30, 2023		67,785,469	$	7	$	488,873		$	(280	)	$	(313,819	)	$	174,781


	Common Stock				Additional Paid-In		Accumulated other comprehensive			Accumulated			Total Stockholders’ Equity
(in thousands, except share amounts)	Shares		Amount		Capital		loss			Deficit			(Deficit)
Balance at December 31, 2021		37,174,741	$	4	$	346,382	$	—		$	(130,123	)	$	216,263
Issuance of common stock upon vesting and exercise of stock options		161,573		—		247		—			—			247
Stock-based compensation expense		—		—		1,746		—			—			1,746
Other comprehensive loss		—		—		—		(1,072	)		—			(1,072	)
Net loss		—		—		—		—			(22,737	)		(22,737	)
Balance at March 31, 2022		37,336,314	$	4	$	348,375	$	(1,072	)	$	(152,860	)	$	194,447
Issuance of common stock upon vesting and exercise of stock options		74,358		—		146		—			—			146
Issuance of common stock from open market sales agreement		95,564		—		553		—			—			553
Stock-based compensation expense		—		—		1,741		—			—			1,741
Other comprehensive loss		—		—		—		(254	)		—			(254	)
Net loss		—		—		—		—			(21,659	)		(21,659	)
Balance at June 30, 2022		37,506,236	$	4	$	350,815	$	(1,326	)	$	(174,519	)	$	174,974
Issuance of common stock upon vesting and exercise of stock options		126,509		—		279		—			—			279
Issuance of common stock from open market sales agreement		760,466		—		3,792		—			—			3,792
Stock-based compensation expense		—				3,329		—			—			3,329
Other comprehensive loss		—		—		—		148			—			148
Net loss		—		—		—		—			(23,788	)		(23,788	)
Balance at September 30, 2022		38,393,211	$	4	$	358,215	$	(1,178	)	$	(198,307	)	$	158,734

The accompanying notes are an integral part of these condensed consolidated financial statements.

Vor Biopharma Inc.

condensed CONSOLIDATED StatementS of Cash Flows

(unaudited)


	Nine Months Ended September 30,
(in thousands)	2023			2022
Cash flows from operating activities
Net loss	$	(91,602	)	$	(68,184	)
Adjustments to reconcile net loss to net cash used in operations:
Depreciation expense		2,600			1,893
Non-cash lease expense		3,513			2,451
Stock-based compensation		11,468			6,816
Interest (amortization) accretion on marketable securities		(3,674	)		163
Changes in operating assets and liabilities:
Operating lease liabilities, net		(2,646	)		(9,037	)
Prepaid expenses and other current assets		2,558			316
Accounts payable and accrued liabilities		2,678			(542	)
Other assets		(1,161	)		(674	)
Net cash used in operating activities		(76,266	)		(66,798	)
Cash flow from investing activities
Purchases of marketable securities		(58,369	)		(5,044	)
Proceeds from maturities of marketable securities		120,000			16,130
Purchases of property and equipment		(958	)		(8,141	)
Net cash provided by investing activities		60,673			2,945
Cash flow from financing activities
Payment of issuance costs related to underwritten public offering and concurrent private placement		(717	)		—
Proceeds from the issuance of common stock from at-the-market sales agreement, net of issuance costs		4,593			4,345
Shares repurchased for tax withholdings upon vesting of restricted stock unit awards		(1,165	)		—
Proceeds from stock option exercises and the issuance of shares under ESPP		202			534
Net cash provided by financing activities		2,913			4,879
Net decrease in cash, cash equivalents and restricted cash equivalents		(12,680	)		(58,974	)
Cash, cash equivalents and restricted cash equivalents, beginning of period	$	60,119		$	122,214
Cash, cash equivalents and restricted cash equivalents, end of period	$	47,439		$	63,240
Supplemental disclosure of non-cash activities
Right-of-use assets obtained in exchange for lease obligations	$	37		$	24,207
Purchases of property and equipment in accounts payable and accrued liabilities	$	63		$	56
Financing costs associated with the sale of common stock included in accounts payable and accrued expenses	$	30		$	—

A reconciliation of the cash, cash equivalents and restricted cash equivalents reported within the condensed consolidated balance sheets that sums to the total of the same amounts shown in the statements of cash flows is as follows:


	September 30,
(in thousands)	2023		2022
Cash and cash equivalents	$	45,026	$	60,827
Restricted cash equivalents		2,413		2,413
Total cash, cash equivalents and restricted cash equivalents as shown on the statements of cash flows	$	47,439	$	63,240

The accompanying notes are an integral part of these condensed consolidated financial statements.

VOR BIOPHARMA INC.

NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS

(UNAUDITED)

1. Nature of the Business

Vor Biopharma Inc. (the “Company”) is a clinical-stage cell and genome engineering company that combines a novel patient engineering approach with targeted therapies to provide a single company solution for patients suffering from hematological malignancies. The Company’s proprietary platform leverages its expertise in hematopoietic stem cell (“HSC”) biology, genome engineering and targeted therapy development to genetically modify HSCs to remove surface targets expressed by cancer cells. The Company is headquartered in Cambridge, Massachusetts. The Company was incorporated on December 30, 2015.

Risks and Uncertainties

The Company is subject to a number of risks common to development stage companies in the biotechnology industry, including, but not limited to, risks of failure of preclinical studies and clinical trials, dependence on key personnel, protection of proprietary technology, reliance on third party organizations, risks of obtaining regulatory approval for any product candidate that it may develop, development by competitors of technological innovations, compliance with government regulations, geopolitical tensions, adverse macroeconomic conditions and the need to obtain additional financing.

The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates. The Company believes that its existing cash, cash equivalents and marketable securities at September 30, 2023 will be sufficient to allow the Company to fund its current operations through at least a period of one year after the date the financial statements are issued.

2. Summary of Significant Accounting Policies

Basis of Presentation

The accompanying condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Certain comparative amounts have been reclassified to conform to the current period presentation. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”).

Use of Estimates

The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the condensed consolidated financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the condensed consolidated financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: accrued expenses and research and development expenses.

Unaudited Interim Financial Information

The condensed consolidated financial statements of the Company included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (the “SEC”). The unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiary. All intercompany transactions and balances have been eliminated in consolidation. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period.

The accompanying condensed consolidated balance sheet as of December 31, 2022 has been derived from the Company’s audited consolidated financial statements for the year ended December 31, 2022. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These unaudited interim condensed consolidated financial statements should be read in conjunction with the audited annual

consolidated financial statements and notes in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 (the “2022 Annual Report”).

During the nine months ended September 30, 2023, there have been no changes to the Company’s significant accounting policies as described in the 2022 Annual Report.

3. Marketable Securities

The amortized cost and estimated fair value of marketable securities, by contractual maturity are as follows:

September 30, 2023

(in thousands)

Amortized Cost

Gross Unrealized Holding Gains

Gross Unrealized Holding Losses

Fair Value

Maturing in one year or less

U.S. Treasuries

115,352